Daratumumab

89 percent of evaluable patients achieved minimal residual disease (MRD) negativity with CARVYKTI® after three-year follow-up in CARTITUDE-4 study; the majority in less than 2 months Results add to the overall survival (OS) benefit recently reported making CARVYKTI® the first and only cell therapy to significantly extend OS versus standard therapies in multiple myeloma Landmark Phase 3 CARTITUDE-4 study data featured as an oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition Dec. 09, 2024 -- Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, announced today new results from the Phase 3 CARTITUDE-4 study that show a single infusion of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) provided significantly higher rates of minimal residual disease (MRD)-negativity in patients with relapsed or lenalidomide-refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD, compared to standard therapies of pomalidomide, bortezomib, and dexamethasone (PVd) or daratumumab, pomalidomide, and dexamethasone (DPd).1 MRD negativity is a prognostic marker of prolonged survival outcomes for patients with multiple myeloma.1 These results reinforce the clinical value of CARVYKTI® as early as second line and support the recent achievement of overall survival (OS) benefit versus standard therapies1. The MRD negativity findings were featured as an oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract #1032) in San Diego, California.1 “The MRD data further underscores the benefits of treatment with CARVYKTI,” said Yi Lin, M.D., Ph.D., hematologist and oncologist at Mayo Clinic, Rochester, MN. “These new findings support CARVYKTI as a transformative therapeutic option, leading to improved progression-free survival, overall survival, and now minimal residual disease negativity.” ‡ The Phase 3 CARTITUDE-4 study evaluated CARVYKTI® in comparison to standard therapies of PVd or DPd for the treatment of adults with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy, including a PI and IMiD, and who were lenalidomide-refractory. In the trial, 208 adults were randomized to receive CARVYKTI®, and 211 to receive standard therapies. The study assessed patients for MRD negativity at the 10-5 threshold (cilta-cel, n=145, standard therapies, n=103). At a median follow-up of almost three years (34 months), evaluable patients treated with CARVYKTI® achieved an MRD-negativity rate of 89% versus 38% for those treated with standard therapies (P® with 69% of MRD-evaluable patients by day 56. At data cutoff, sustained MRD-negative ≥CR of at least 12 months was achieved in 52% of MRD-evaluable patients in the CARVYKTI® arm vs. 10% in the standard of care arm (P® was administered earlier in the treatment regimen. “The latest MRD data showcases the advances of CARVYKTI and further demonstrates why it is a leading treatment for patients with multiple myeloma,” said Ying Huang, Ph. D., Chief Executive Officer of Legend Biotech. “As we strive to transform the therapeutic landscape in cancer and beyond, we are proud of the progress made and will continue our efforts to improve the quality of life for those battling incurable diseases.” Data from CARTITUDE-4 supported the U.S. Food and Drug Administration (FDA) and European Commission (EC) approval of CARVYKTI® earlier this year for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a PI, and IMiD, and are refractory to lenalidomide.1 CARVYKTI® is the first and only BCMA-targeted CAR-T cell therapy approved for the treatment of patients with multiple myeloma who have had at least one prior line of therapy. Globally, CARVYKTI® is now commercially available in five countries and has been utilized by over 4,500 patients. Ciltacabtagene autoleucel is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA. The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.2 In December 2017, Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen), a Johnson & Johnson company, to develop and commercialize cilta-cel. In February 2022, cilta-cel was approved by the U.S. Food and Drug Administration (FDA) under the brand name CARVYKTI® for the treatment of adults with relapsed or refractory multiple myeloma. In April 2024, cilta-cel was approved for the second-line treatment of patients with relapsed/refractory myeloma who have received at least one prior line of therapy including a proteasome inhibitor, an immunomodulatory agent, and are refractory to lenalidomide. In May 2022, the European Commission (EC) granted conditional marketing authorization of CARVYKTI® for the treatment of adults with relapsed or refractory multiple myeloma. In September 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI®. Cilta-cel was granted Breakthrough Therapy Designation in the U.S. in December 2019 and in China in August 2020. In addition, cilta-cel received a PRIority MEdicines (PRIME) designation from the European Commission in April 2019. Cilta-cel also received Orphan Drug Designation from the U.S. FDA in February 2019, from the European Commission in February 2020, and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In March 2022, the European Medicines Agency’s Committee for Orphan Medicinal Products recommended by consensus that the orphan designation for cilta-cel be maintained on the basis of clinical data demonstrating improved and sustained complete response rates following treatment. CARTITUDE-4 (NCT04181827) is an ongoing, international, randomized, open-label Phase 3 study evaluating the efficacy and safety of cilta-cel versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) in adult patients with relapsed or lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy, including a PI and an IMiD.3 Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.4 In 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the U.S.5 While some patients with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.6 Legend Biotech is a global biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogeneic chimeric antigen receptor T-cell, gamma-delta T cell (gd T) and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of cutting-edge therapeutics for patients worldwide. REFERENCES ____________________________________ 1 Rakesh Popat et al. Ciltacabtagene Autoleucel (Cilta-cel) Vs Standard of Care (SoC) in Patients with Lenalidomide (Len)-Refractory Multiple Myeloma (MM) after 1-3 Lines of Therapy: Minimal Residual Disease (MRD) Negativity in the Phase 3 Cartitude-4 Trial. American Society of Hematology 2024 Annual Meeting. December 2024 2 CARVYKTI™ Prescribing Information. Horsham, PA: Janssen Biotech, Inc. 3 ClinicalTrials.Gov. A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma (CARTITUDE-4). https://www.clinicaltrials.gov/study/NCT04181827. Accessed March 2024. 4 American Cancer Society. ”What is Multiple Myeloma?”. Available at: https://www.cancer.org/cancer/types/multiple-myeloma/about/what-is-multiple-myeloma.html. Accessed March 2024. 5 American Cancer Society. “Key Statistics About Multiple Myeloma.” Available at: https://www.cancer.org/cancer/types/multiple-myeloma/about/key-statistics.html. Accessed March 2024 6 American Cancer Society. Multiple myeloma: early detection, diagnosis, and staging. Available at: https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf. Accessed March 2023. 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The Institute for Clinical and Economic Review on Thursday released a new report arguing that US healthcare costs rose by more than $800 million from price increases on just five drugs’ net prices, hikes that weren’t backed by new clinical evidence. The five drugs with these “unsupported price increases,” ICER says, include Gilead’s HIV/AIDS drug Biktarvy, J&J’s multiple myeloma drug Darzalex, Novartis’ heart drug Entresto, which is one of the first drugs under government price negotiation in 2026, Exelixis’ cancer drug Cabometyx, and Pfizer’s Xeljanz, which can treat inflammatory and autoimmune conditions. To select the top 10, the group looked at 250 drugs, chosen because of their 2023 net sales revenue. It also flagged five drugs with new evidence backing their use that didn’t make the “unsupported” list, but emphasized that those “should not be interpreted to mean that the new evidence justifies the level of price increase; a full cost-effectiveness assessment was not conducted to determine the comparative value.” The topic, and ICER’s list, routinely generate pushback from drug companies that disagree with how the review is conducted. The National Pharmaceutical Council, a drug industry trade group, argued that the reports “do more harm than good: They have serious methodological limitations, contradict ICER’s core value assessment framework, and ignore a tremendous amount of evidence submitted by manufacturers.” Kimberly Westrich, NPC chief strategy officer, told Endpoints News by email, “We continue to call on ICER to stop producing this consistently flawed report that misleads stakeholders and risks driving harmful policy decisions.”